💊#FDA Approval Update | #Avlayah: the first therapy targeting neurological symptoms of Hunter syndrome
🧠 Designed to cross the BBB, addressing a major unmet need in this #RareDisease
📉 ~91% reduction in #CSF heparan sulfate in trials
🧪MCE provides tividenofusp alfa for research use
#HunterSyndrome

FDA clears Denali's 'game-changer' Hunter syndrome drug Denali's Avlayah is the first drug for neurological complications associated Hunter syndrome to be approved in the US.

#Denali's Avlayah is the first drug for neurological complications associated #Huntersyndrome to be approved in the US.

pharmaphorum.com/news/fda-cle...

New in JIMD Reports:
Open-label phase 2/3 substudy of intrathecal idursulfase-IT in children <3 years with neuronopathic MPS II.
Reports long-term follow-up with generally stable cognitive assessments and reduced CSF GAG levels in a small cohort.
🔗 doi.org/10.1002/jmd2...
#MPSII #HunterSyndrome# IMD

Boy with Rare Genetic Disorder Amazes Doctors After World-First Gene Therapy The first child in history has successfully been treated with a new genetic therapy for an ultra-rare developmental defect called Hunter syndrome. Several years in the making, Oliver Chu became the first in the world to receive the stem cell-based treatment in February, and 3 months later seemed to be a normal child again, meeting […] The post Boy with Rare Genetic Disorder Amazes Doctors After World-First Gene Therapy appeared first on Good News Network.

Boy with Rare Genetic Disorder Amazes Doctors After World-First Gene Therapy: The first child in history has successfully been treated with a new genetic therapy for an ultra-rare developmental… @goodnewsnetwork.org #GeneticDisorder #GeneTherapy #HunterSyndrome #MedicalBreakthrough #RareDisease

This Toddler Was the First Person to Receive a Test Treatment for a Rare Genetic Disorder. Nine Months Later, His Progress Is Inspiring Hope Oliver Chu is one of five kids participating in a clinical trial investigating a gene therapy for Hunter syndrome, a disorder with symptoms akin to childhood dementia that limits life expectancy

This Toddler Was the First Person to Receive a Test Treatment for a Rare Genetic Disorder. Nine Months Later, His Progress Is Inspiring Hope: Oliver Chu is one of five kids participating in a clinical trial… @SmithsonianMag #RareDisease #GeneTherapy #HunterSyndrome #ChildHealth #ClinicalTrials

Hunter syndrome: Boy with rare condition amazes doctors after world-first gene therapy Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.

Hunter’s and Hurler’s syndromes both characteristically lead to hearing loss and we are pleased to see this breakthrough for the children we are helping to monitoring for this study at the Royal Manchester Children’s Hospital, Clinical Research Facility.
#RareDisease #HunterSyndrome #HurlerSyndrome

🧪🔬Boy with rare condition amazes doctors after world-first gene therapy | BBC News

BBC News 11/25/2025
www.youtube.com/watch?v=71vG...

#GeneTherapy
#Science
#HunterSyndrome
#MPS2

Please watch and forward. Share with family at Thanksgiving table so more people know about this.

www.youtube.com/watch?v=71vG...
Engaging, accessible & informative
7 & 1/2 min video tells the story of precious Oliver, the 1st #MPSII #HunterSyndrome recipient of a groundbreaking #gene-therapy treatment.

Hunter syndrome: Boy with rare condition amazes doctors after world-first gene therapy Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.

A 3-year-old boy appears to have fully recovered after receiving the first-ever #GeneTherapy to treat #HunterSyndrome earlier this year, researchers revealed yesterday.
www.bbc.com/news/article...

From California to Manchester: How One Gene Therapy Is Rewriting Hunter Syndrome Care - Biotech Industry Examiner Hunter syndrome – or mucopolysaccharidosis type II (MPS II) – is the kind of diagnosis that collapses a family’s sense of time.

A 3-year-old from California flies to Manchester for gene therapy.

Our new piece on how rare-disease care is becoming “passport medicine”.
🔗 biotech.industryexaminer.com/passport-med...

#biotech #genetherapy #raredisease #HunterSyndrome #MPSII #GlobalHealth #HealthPolicy #TechNews

Hunter syndrome: Boy with rare condition amazes doctors after world-first gene therapy Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.

#OliverChu has a rare, inherited condition called #Huntersyndrome or #MPSII which causes progressive damage to the body and brain. In the most severe cases, patients with the disease usually die before the age of 20, sometimes described as a type of childhood #dementia.
www.bbc.com/news/article...

Hunter syndrome: Boy with rare condition amazes doctors after world-first gene therapy Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.

Prof Simon Jones, who is co-leading the trial tells the BBC: "I've been waiting 20 years to see a boy like Ollie doing as well as he is, and it's just so exciting." #HunterSyndrome almost always occurs in boys affecting one in 100,000. 👨‍⚕️ #ScienceMatters #GeneTherapy 🧬

www.bbc.com/news/article...

Hunter syndrome: Boy with rare condition amazes doctors after world-first gene therapy **A three-year-old boy has astounded doctors with his progress after becoming the first person in the world with his devastating disease to receive a ground-breaking gene therapy.** Oliver Chu has a rare, inherited condition called Hunter syndrome - or MPSII - which causes progressive damage to the body and brain. Due to a faulty gene, before the treatment Oliver was unable to produce an enzyme crucial for keeping cells healthy. In a world first, medical staff in Manchester have tried to halt the disease by altering Oliver's cells using gene therapy.

In a world-first, a young boy with rare Hunter syndrome is absolutely thriving after receiving groundbreaking gene therapy! Doctors are amazed by his incredible progress, offering new hope for many. ✨ #HunterSyndrome #GeneTherapy #MedicalBreakthrough #UpliftingNews

Three-Year-Old Thrives After World-First Gene Therapy for Rare Hunter Syndrome World-First Gene Therapy Shows Promising Results in Hunter Syndrome A three-year-old boy from California, Oliver Chu, has amazed medical experts

Three-year-old Oliver Chu shows remarkable recovery after receiving the first-ever gene therapy for Hunter syndrome.

#HunterSyndrome #GeneTherapy #MedicalBreakthrough #ChildHealth #PediatricInnovation

New method ➡️ Domain-substituted IGF2 tag modulates targeting of lentiviral #GeneTherapy for #HunterSyndrome

By F. Catalano, WWMP Pijnappel & colleagues @erasmusmc.bsky.social

🗞️ doi.org/10.1038/s443...

Neuroscience Weekly News – August 6th 2025 🧠 This Week in Neuroscience: MS Genetics Breakthrough, $372.5M CNS Funding, AI for Drug Discovery, and More Explore the latest […] The post Neuroscience Weekly News – August 6th 2025 appeared first on LucidQuest Ventures.

FYI: LucidQuest Views >>> Neuroscience Weekly News – August 6th 2025 #News #EpilepsyResearch #genetherapy #HunterSyndrome Comment below!

Neuroscience Weekly News – August 6th 2025 🧠 This Week in Neuroscience: MS Genetics Breakthrough, $372.5M CNS Funding, AI for Drug Discovery, and More Explore the latest […] The post Neuroscience Weekly News – August 6th 2025 appeared first on LucidQuest Ventures.

ICYMI: LucidQuest Views >>> Neuroscience Weekly News – August 6th 2025 #News #EpilepsyResearch #genetherapy #HunterSyndrome Comment below!

Neuroscience Weekly News – August 6th 2025 🧠 This Week in Neuroscience: MS Genetics Breakthrough, $372.5M CNS Funding, AI for Drug Discovery, and More Explore the latest […] The post Neuroscience Weekly News – August 6th 2025 appeared first on LucidQuest Ventures.

LucidQuest Views >>> Neuroscience Weekly News – August 6th 2025 #News #EpilepsyResearch #genetherapy #HunterSyndrome Comment below!

More options for treating individuals with MPS II? Idursulfase beta is safe and effective for the management of #HunterSyndrome bit.ly/4kt5pM7 #Mucopolysaccharidosis

I’m jumping into a freezing cold lake to help give my son a chance to grow up. Support my cause today so we can find a cure for Hunter syndrome.

polarotary.rallyup.com/3e4e24/m/kri...

#HunterSyndrome #MPSII #RareDisease #LakeArrowhead #ProjectAlive #Charity #PolarPlunge

BCBS of Tennessee wants my son to suffer so they can save a little green. This is the system. And shaming them is the only thing that's ever worked to make things happen.
Friends - make your calls, post your advocacy, whatever you can do to help. #huntersyndrome

www.instagram.com/reel/DDxfZDD...